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Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders

Primary Purpose

Mitochondrial Myopathies, Fatty Acid Oxidation Defects

Status

Completed

Phase

Not Applicable

Locations

Denmark

Study Type

Interventional

Intervention

Resveratrol

About this trial

This is an interventional treatment trial for Mitochondrial Myopathies

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patient is willing and able to provide written informed consent prior to participation.
Patient is ≥18 and ≤80 years of age at baseline.
Patients have genetically verified mitochondrial disorder or a fatty acid oxidation deficiency (VLCAD/CPTII).
Patient has a clinical presentation, signs or symptoms suggestive of myopathy (e.g., easy fatigability, exercise intolerance, muscle pain) in the opinion of the Investigator.
Patient is ambulatory.

Exclusion Criteria:

Patient has any prior or current medical conditions that, in the judgment of the Investigator, would prevent the patient from safely participating in and/or completing all study requirements.
Patient has symptoms of mitochondrial myopathy due to known secondary mitochondrial dysfunction (e.g., drug-induced myopathy).
Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments.
Pregnancy or breastfeeding

Sites / Locations

Copenhagen Neuromuscular Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

Resveratrol

Arm Description

Over the counter supplement

Outcomes

Primary Outcome Measures

Heart rate

Decrease in heart rate during constant load cycling exercise.

Secondary Outcome Measures

Peak oxygen utilization

VO^2max (ml/min)

Fatty acid oxidation

Fatty acid oxidation will be assessed by stable isotope technique (only for fatty acid oxidation defect disease subgroup)

Perceived exertion

Evaluation of perceived exertion (Borg score) during constant workload cycling

Fatigue Severity Scale score

Evaluation of self-rated fatigue

SF-36 questionnaire

Evaluation of self-rated daily function scores

Full Information

NCT ID

NCT03728777

First Posted

April 19, 2018

Last Updated

November 26, 2019

Sponsor

Rigshospitalet, Denmark

1. Study Identification

Unique Protocol Identification Number

NCT03728777

Brief Title

Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders

Official Title

Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders: A Double-blind, Placebo-controlled, Cross Over Study

Study Type

Interventional

2. Study Status

Record Verification Date

November 2019

Overall Recruitment Status

Completed

Study Start Date

April 9, 2018 (Actual)

Primary Completion Date

October 1, 2019 (Actual)

Study Completion Date

October 1, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Rigshospitalet, Denmark

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

The purpose of this study is to investigate the potential beneficial effects of a daily supplement of Resveratrol (1000mg/day) on physical ability and on muscle metabolism in patients with verified mitochondrial myopathy and patients with a verified fatty acid oxidation defect of VLCAD and CPTII deficiencies. Investigators hypothesize an improved muscle metabolism, mitochondrial function, fatty acid oxidation and thus improvement of physical ability.

Detailed Description

Study design: double-blind, randomized, placebo-controlled, cross-over study. To ensure enough participants, and due to the risk of a heterogeneous cohort, a cross-over design is chosen, where participants are their own controls. 10 patients with mitochondrial myopathy and 10 patients with fatty acid oxidation defects will be included. Eligible patients will be randomized using a 1:1 assignment ratio to receive placebo or RSV first. Each treatment will be administered orally twice daily for 8 weeks, followed by a 4 weeks wash-out, and afterwards a new 8-week treatment period. During the 20-week trial period, subjects will visit the trial site on five occasions, for functional assessments (cycle ergometer testing), blood sampling and questionnaires.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Mitochondrial Myopathies, Fatty Acid Oxidation Defects

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Crossover Assignment

Model Description

double-blind, randomized, placebo-controlled, cross-over study.

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Title

Resveratrol

Arm Type

Experimental

Arm Description

Over the counter supplement

Intervention Type

Dietary Supplement

Intervention Name(s)

Resveratrol

Intervention Description

Resveratrol supplementation 1000 mg /day or placebo

Primary Outcome Measure Information:

Title

Heart rate

Description

Decrease in heart rate during constant load cycling exercise.

Time Frame

20 weeks

Secondary Outcome Measure Information:

Title

Peak oxygen utilization

Description

VO^2max (ml/min)

Time Frame

20 weeks

Title

Fatty acid oxidation

Description

Fatty acid oxidation will be assessed by stable isotope technique (only for fatty acid oxidation defect disease subgroup)

Time Frame

20 weeks

Title

Perceived exertion

Description

Evaluation of perceived exertion (Borg score) during constant workload cycling

Time Frame

20 weeks

Title

Fatigue Severity Scale score

Description

Evaluation of self-rated fatigue

Time Frame

20 weeks

Title

SF-36 questionnaire

Description

Evaluation of self-rated daily function scores

Time Frame

20 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patient is willing and able to provide written informed consent prior to participation. Patient is ≥18 and ≤80 years of age at baseline. Patients have genetically verified mitochondrial disorder or a fatty acid oxidation deficiency (VLCAD/CPTII). Patient has a clinical presentation, signs or symptoms suggestive of myopathy (e.g., easy fatigability, exercise intolerance, muscle pain) in the opinion of the Investigator. Patient is ambulatory. Exclusion Criteria: Patient has any prior or current medical conditions that, in the judgment of the Investigator, would prevent the patient from safely participating in and/or completing all study requirements. Patient has symptoms of mitochondrial myopathy due to known secondary mitochondrial dysfunction (e.g., drug-induced myopathy). Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments. Pregnancy or breastfeeding

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

John Vissing, professor

Organizational Affiliation

Copenhangen Neuromuscular Center, Rigshospitalet, Denmark

Official's Role

Study Director

Facility Information:

Facility Name

Copenhagen Neuromuscular Center

City

Copenhagen

ZIP/Postal Code

2100

Country

Denmark

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Learn more about this trial

Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders

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